Editor’s Note: Elizabeth Yuko, Ph.D., is a bioethicist and writer whose work has appeared in The New York Times, Rolling Stone, The Washington Post, The Atlantic and elsewhere. She is also an adjunct professor at Fordham University. The views expressed here are hers. Read more opinion on CNN.In December, the US Food and Drug Administration (FDA) approved the first two cell-based gene therapies for treating sickle cell disease (SCD) in people ages 12 and older: Casgevy and Lyfgenia. Since then, questions and concerns have abounded about the cost and accessibility of these treatments — including from Health and Human Services Secretary Xavier Becerra, who has consistently highlighted the financial barriers standing between people living with SCD and these new gene therapies.Elizabeth Yuko – Karen NovakWhile both Casgevy and Lyfgenia are breakthroughs, Casgevy is important for one more reason: It’s the first FDA-cleared clinical use of CRISPR-Cas9 (commonly just referred to as CRISPR) gene editing for the treatment of any condition. (Though Lyfgenia also relies on gene modification, it uses different, better-established technology.)Gene editing refers to various methods that can be used to introduce, replace or remove DNA sequences in the genomes of organisms — including humans. CRISPR acts like genetic scissors to cut and then edit DNA in a particular spot, and has gained favor among researchers for being easier, faster, more accurate and less expensive than earlier techniques.This first FDA-approved therapeutic application of CRISPR is, without question, a significant step forward in the field of gene therapy and the practice of medicine. And, like any emerging medical technology, the initial use of the innovative gene editing tool has been met with an amalgam of anticipation and anxiety.To be sure, there are ethical issues at play — most notably, questions of accessibility of the new multimillion-dollar treatments. But as the gene editing tool becomes part of the toolkit for treating an increasing number of conditions, it’s important not to conflate or even associate this application of CRISPR with more problematic uses of the technology.Casgevy and Lyfgenia are long overdueThe group of inherited red blood cell disorders collectively known as SCD is characterized by abnormalities that make a person’s red blood cells sticky and C-shaped instead of round. These sickle cells cause blockages in a patient’s blood vessels, often resulting in pain and other complications.The US Centers for Disease Control and Prevention estimates that it affects approximately 100,000 people in the United States. SCD is most prevalent among those with African ancestry, according to the National Heart, Lung, and Blood Institute. A study published in May 2023 indicates that of the nearly 75,000 people hospitalized for SCD in the United States between 2016 and 2018, 93% were Black. In the US, about 20,000 people are thought to have a form of the disease severe enough to potentially qualify for these gene-based treatments.Abou …
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[mwai_chat context=”Let’s have a discussion about this article:nnEditor’s Note: Elizabeth Yuko, Ph.D., is a bioethicist and writer whose work has appeared in The New York Times, Rolling Stone, The Washington Post, The Atlantic and elsewhere. She is also an adjunct professor at Fordham University. The views expressed here are hers. Read more opinion on CNN.In December, the US Food and Drug Administration (FDA) approved the first two cell-based gene therapies for treating sickle cell disease (SCD) in people ages 12 and older: Casgevy and Lyfgenia. Since then, questions and concerns have abounded about the cost and accessibility of these treatments — including from Health and Human Services Secretary Xavier Becerra, who has consistently highlighted the financial barriers standing between people living with SCD and these new gene therapies.Elizabeth Yuko – Karen NovakWhile both Casgevy and Lyfgenia are breakthroughs, Casgevy is important for one more reason: It’s the first FDA-cleared clinical use of CRISPR-Cas9 (commonly just referred to as CRISPR) gene editing for the treatment of any condition. (Though Lyfgenia also relies on gene modification, it uses different, better-established technology.)Gene editing refers to various methods that can be used to introduce, replace or remove DNA sequences in the genomes of organisms — including humans. CRISPR acts like genetic scissors to cut and then edit DNA in a particular spot, and has gained favor among researchers for being easier, faster, more accurate and less expensive than earlier techniques.This first FDA-approved therapeutic application of CRISPR is, without question, a significant step forward in the field of gene therapy and the practice of medicine. And, like any emerging medical technology, the initial use of the innovative gene editing tool has been met with an amalgam of anticipation and anxiety.To be sure, there are ethical issues at play — most notably, questions of accessibility of the new multimillion-dollar treatments. But as the gene editing tool becomes part of the toolkit for treating an increasing number of conditions, it’s important not to conflate or even associate this application of CRISPR with more problematic uses of the technology.Casgevy and Lyfgenia are long overdueThe group of inherited red blood cell disorders collectively known as SCD is characterized by abnormalities that make a person’s red blood cells sticky and C-shaped instead of round. These sickle cells cause blockages in a patient’s blood vessels, often resulting in pain and other complications.The US Centers for Disease Control and Prevention estimates that it affects approximately 100,000 people in the United States. SCD is most prevalent among those with African ancestry, according to the National Heart, Lung, and Blood Institute. A study published in May 2023 indicates that of the nearly 75,000 people hospitalized for SCD in the United States between 2016 and 2018, 93% were Black. In the US, about 20,000 people are thought to have a form of the disease severe enough to potentially qualify for these gene-based treatments.Abou …nnDiscussion:nn” ai_name=”RocketNews AI: ” start_sentence=”Can I tell you more about this article?” text_input_placeholder=”Type ‘Yes'”]
